Berotralstat for the prophylaxis of hereditary angioedema—Real‐world evidence data from the United Kingdom

Ahuja, Manisha; Dorr, Anthony D.; Bode, Eniola; Boulton, Anne Pacita Rosillo; Buckland, Matthew; Chee, Samuel; Dalley, Claire; Denman, Sarah; Ekbote, Anjali; Elkhalifa, Shuayb; El‐Shanawany, Tariq; Eren, Erdem; Herwadkar, Archana; Garcez, Tomaz; Ghanta, Harichandana; Grammatikos, Alexandros P.; Grigoriadou, Sofia; Jain, Rashmi; Lorenzo, Lorena; Manson, Ania; Moon, Emily Haejoon; Murng, Sai; Murphy, Aveen; Mutlu, Leman; Peters, Nicholas; Sooriyakumar, Kavitha; Stroud, Catherine B.; Townsend, Katie; Yellon, Robert L.; Yong, Patrick; Kiani‐Alikhan, Sorena

doi:10.1111/all.15641

Cited by 8 publications

(7 citation statements)

References 5 publications

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“… The mean AECT score for HAE type I patients ( n = 7) at baseline was 6.1; this increased to 10.0 after 3 months of treatment, and to 13.3 after 6 months of treatment. This confirms results reported from other studies concerning real-world data [ 7 ]. After the 6-month interval, a slight drop in scores was noticed, which may be due to a subjective decrease in satisfaction as patients strive for less to no attacks and therefore satisfaction decreases again, which is reflected in the questionnaires.…”

Section: Discussionsupporting

confidence: 93%

“…Real-world data concerning treatment with Berotralstat is limited [ 7 , 8 ]. AAE-C1-INH is a rare disease and very few studies have been performed to date examining the use of LTP in patients with AAE-C1-INH.…”

Section: Discussionmentioning

confidence: 99%

“…The most common adverse effects include headaches as well as gastrointestinal complaints including abdominal pain and diarrhea. Published real-world evidence concerning Berotralstat is limited [ 7 , 8 ]. A comparative meta-analysis juxtaposing prophylactic treatment using Lanadelumab given once every 2 or 4 weeks versus Berotralstat concluded that Lanadelumab has a superior effectiveness in reducing attacks within 28 days [ 9 ].…”

Section: Introductionmentioning

confidence: 99%

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A Retrospective Analysis of Long-Term Prophylaxis with Berotralstat in Patients with Hereditary Angioedema and Acquired C1-Inhibitor Deficiency—Real-World Data

Johnson,

Stenzl,

Hofauer

et al. 2023

Clinic Rev Allerg Immunol

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Hereditary angioedema (HAE) and acquired C1-inhibitor deficiency (AAE-C1-INH) are orphan diseases. Berotralstat is a recently licensed long-term prophylaxis (LTP) and the first oral therapy for HAE patients. No approved therapies exist for AAE-C1-INH patients. This study is the first to report real-world clinical data of patients with AAE-C1-INH and HAE who received Berotralstat. All patients treated with Berotralstat were included in this retrospective, bi-centric study. Data was collected from patients’ attack calendars and the angioedema quality of life (AE-QoL) and angioedema control test (AECT) questionnaires before treatment, and at 3, 6, and 12 months after treatment and was then analyzed. Twelve patients were included, 3 patients with AAE-C1-INH, 7 patients with HAE type I, and 2 patients with HAE-nC1-INH. One patient (HAE I) quit treatment. Berotralstat was associated with fewer attacks in all groups. After 6 months of treatment, a median decrease of attacks per month was noted for HAE type I patients (3.3 to 1.5) and AAE-C1-INH patients (2.3 to 1.0). No aerodigestive attacks were noted for AAE-C1-INH patients. For HAE-nC1-INH patients, a mean decrease from 3.8 to 1.0 was noted (3 months). For HAE I patients, the total AE-QoL lowered a mean of 24.1 points after 6 months, for HAE-nC1-HAE patients 8.0 points, and for AAE-C1-INH patients 13.7 points. AECT scores increased for HAE I patients (mean: 7.1), HAE-nC1-INH patients (9.0), and AAE-C1-INH patients (4.2) after 6 months. Patients with HAE, HAE-nC1-INH, and AAE-C1-INH treated with Berotralstat showed reduced angioedema attacks and improved AE-QoL and AECT scores.

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Section: Discussionsupporting

confidence: 93%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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A Retrospective Analysis of Long-Term Prophylaxis with Berotralstat in Patients with Hereditary Angioedema and Acquired C1-Inhibitor Deficiency—Real-World Data

Johnson,

Stenzl,

Hofauer

et al. 2023

Clinic Rev Allerg Immunol

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“…Our findings are similar to the results of real-world studies from other jurisdictions. A study from the United Kingdom (UK) reported effectiveness and adverse effects in 54 patients using patient surveys administered at one time point (with a recall period of up to 9 months) ( 11 ). They found a 64.9% reduction in attack frequency 4-6 months after starting berotralstat.…”

Section: Discussionmentioning

confidence: 99%

Berotralstat in hereditary angioedema due to C1 inhibitor deficiency: first real-world evidence from a Canadian center

Srinivasan,

Ritchie,

Adatia

2024

Front. Immunol.

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BackgroundHereditary angioedema due to C1 inhibitor deficiency is a rare genetic condition that causes recurrent swelling with consequent functional impairment and decreased quality of life. Long-term prophylaxis (LTP) to prevent angioedema episodes is a key component of disease management. Berotralstat, an oral, once-daily plasma kallikrein inhibitor, was approved for LTP by Health Canada in 2022.MethodsWe conducted a retrospective, real-world study investigating the effectiveness and adverse effects of berotralstat. Data on angioedema frequency, disease control, and adverse events were tabulated. Patient satisfaction with treatment was scored on a 5-point Likert scale, with 1 representing very unsatisfied and 5 representing very satisfied with therapy.ResultsFrom June, 2022 and May, 2023, 8 patients with HAE type 1 or type 2 received berotralstat. Effectiveness data were available for 7 patients who continued the drug for at least 3 months, 4 of whom switched to berotralstat from plasma-derived C1 inhibitor LTP. In these 7 patients, the average number of attacks per month decreased from 3.3 to 1.6 (p<0.05), representing a ~52% reduction in attack frequency. Median angioedema control test score numerically improved from 8 to 13 (p=0.0781). Of the 8 patients who received berotralstat, 3 reported no adverse effects and 5 experienced gastrointestinal side effects, which were mild and transient in 3 and led to discontinuation in 1. Average treatment satisfaction was between satisfied and very satisfied at 4.3.ConclusionBerotralstat is an effective agent for long-term prophylaxis in HAE. Most patients experienced no adverse effects or mild, transient gastrointestinal symptoms.

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“…There is clear evidence from clinical trials and real-world data that licenced LTPs are more efficacious and better tolerated than AAs, 1,17 and this is reassuring for patients, encouraging them to switch treatments.…”

Section: Encouraging More People To Transition From Second-to First-l...mentioning

confidence: 99%

Transitioning Patients From Second- to First-Line Prophylaxis in Hereditary Angioedema

Cross

2024

EMJ

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Berotralstat for the prophylaxis of hereditary angioedema—Real‐world evidence data from the United Kingdom

Cited by 8 publications

References 5 publications

A Retrospective Analysis of Long-Term Prophylaxis with Berotralstat in Patients with Hereditary Angioedema and Acquired C1-Inhibitor Deficiency—Real-World Data

A Retrospective Analysis of Long-Term Prophylaxis with Berotralstat in Patients with Hereditary Angioedema and Acquired C1-Inhibitor Deficiency—Real-World Data

Berotralstat in hereditary angioedema due to C1 inhibitor deficiency: first real-world evidence from a Canadian center

Transitioning Patients From Second- to First-Line Prophylaxis in Hereditary Angioedema

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